[Reporter's View] Disparity btwn approval·reimb criteria for cancer drugs

Recently, there has been a discrepancy between the scope of approval and health insurance reimbursement criteria in cancer treatment. Consequently, there are growing cases in which patients who are technically eligible for treatment under the approved indication are unable to achieve therapeutic benefits.

Critics point out that the widening gap among global clinical guidelines, real-world clinical practice, and domestic regulatory approval and reimbursement criteria is becoming yet another barrier to patient care.

The aims of regulatory approval and reimbursement are inherently different. Approval is a process of evaluating therapeutic viability based on safety and efficacy, whereas reimbursement evaluates both cost-effectiveness and clinical necessity within a constrained healthcare budget. Consequently, the criteria of these categories are different. However, instances where the approved label, global guideline recommendations, and actual reimbursement scope diverge are increasingly common, aggravating confusion in clinical settings.

One of these examples is the treatment of advanced renal cell carcinoma (RCC).

Global clinical guidelines offer a broader range of therapeutic choices following first-line immuno-oncology therapy for RCC.

Major international guidelines, including the National Comprehensive Cancer Network (NCCN), European Society for Medical Oncology (ESMO), and European Association of Urology (EAU), recommend various targeted therapies as key subsequent lines of therapy after immuno-oncology combination therapies. In particular, certain agents are highlighted as preferred choices after first-line therapy, suggested as a flexible, sequential treatment strategy depending on patient status and therapeutic response.

However, real-world clinical practices in South Korea are quite different. The approved scope for several subsequent treatment options is limited to prior exposure to VEGF-targeted therapy. Consequently, for patient cohorts who received first-line immuno-oncology combination therapy, utilization is restricted, and reimbursement is entirely unavailable. While global guidelines are expanding therapeutic choices, a restrictive structure remains, where approval and reimbursement criteria narrow down the eligible patient population.

This gap is also evident in the treatment of metastatic gastric cancer. Based on evidence of survival benefits, immune-oncology combination therapy has recently become a key pillar of treatment, with some agents securing regulatory approval for an all-comer patient population. At the reimbursement stage, however, the target patient population is redefined by reflecting the biomarker thresholds based on PD-L1 expression levels, such as Combined Positive Score (CPS) or Tumor Area Positivity (TAP).

As a result, certain eligible patients are excluded from reimbursement coverage. In real-world clinical practice, even when a physician determines a clear clinical necessity for treatment, considering patients whose biomarker scores are near the cut-off threshold or who have an overall poor performance status, therapeutic choices are limited by rigid reimbursement rules. This indicates that the discrepancy between regulatory approval and reimbursement guidelines directly lead to disparities in patient access to treatments.

Of course, it is challenging to extend reimbursement coverage to every therapy based on its approved scope. Balancing fiscal sustainability and cost-effectiveness in the national health insurance budget is necessary.

Selecting specific patient populations and establishing prioritization within limited financial resources are necessary steps. The critical issue, however, is the degree of the gap between these criteria and actual clinical practice. If global guidelines and regulatory frameworks are designed to expand access to treatment while reimbursement criteria remain anchored in outdated patient-selection frameworks, the lag between shifting therapeutic paradigms and institutional policies will inevitably widen.

If regulatory approval is the process of unlocking therapeutic potential, reimbursement is the process that translates that potential into clinical reality. A certain degree of variance between the two criteria can exist. However, when the gap widens to the point of gauging patient access to life-saving treatments, it is time to re-evaluate what the system is missing.